Observational Study
Copyright ©The Author(s) 2023. Published by Baishideng Publishing Group Inc. All rights reserved.
World J Gastrointest Surg. Nov 27, 2023; 15(11): 2537-2552
Published online Nov 27, 2023. doi: 10.4240/wjgs.v15.i11.2537
Multi-national observational study to assess quality of life and treatment preferences in patients with Crohn’s perianal fistulas
Chitra Karki, Amod Athavale, Vijay Abilash, Gary Hantsbarger, Parnia Geransar, Kate Lee, Slobodan Milicevic, Marko Perovic, Leanne Raven, Magdalena Sajak-Szczerba, Abigail Silber, Annabelle Yoon, Phil Tozer
Chitra Karki, Global Evidence and Outcomes-Gastroenterology, Takeda Pharmaceuticals United States, Inc, Cambridge, MA 02139, United States
Amod Athavale, Vijay Abilash, Abigail Silber, Trinity Partners, LLC, Waltham, MA 02451-7528, United States
Gary Hantsbarger, Observational Research, Takeda Pharmaceuticals United States, Inc, Cambridge, MA 02139, United States
Parnia Geransar, Slobodan Milicevic, Medical Affairs, Takeda Pharmaceuticals International Co., Opfikon 8152, Zurich, Switzerland
Kate Lee, Research and Patient Programs, Crohn’s and Colitis Canada, 600-60 St. Clair Avenue East, Toronto M4T 1N5, Ontario, Canada
Marko Perovic, Treasurer, European Federation of Crohn’s & Ulcerative Colitis Associations, Brussels B 1000, Belgium
Leanne Raven, Crohn’s and Colitis Australia, Camberwell South, VIC 3124, Australia
Magdalena Sajak-Szczerba, European Federation of Crohn’s & Ulcerative Colitis Associations, Brussels B 1000, Belgium
Annabelle Yoon, Japan Medical Office, Takeda Pharmaceutical Company Limited, Tokyo 103-8668, Japan
Phil Tozer, Department of Colorectal Surgery, St Mark’s Hospital and Academic Institute, London HA1 3UJ, United Kingdom
Author contributions: Karki C, Athavale A, Abilash V, Hantsbarger G, Geransar P, Lee K, Milicevic S, Perovic M, Raven L, Sajak-Szczerba M, Silber A, Yoon A, and Tozer P contributed to the conceptualization of the study; Athavale A, Abilash V, and Silber A contributed to the data curation; Athavale A, Abilash V, and Silber A contributed to the formal analysis; Karki C contributed to the funding acquisition; Karki C, Athavale A, Abilash V, and Silber A contributed to the investigation; Karki C, Athavale A, Abilash V, Hantsbarger G, and Tozer P performed the methodology; Athavale A, Abilash V, and Silber A contributed to the project administration; Karki C and Athavale A contributed to the resourcing; Athavale A provided software expertise; Karki C and Athavale A contributed to the supervision of the study; Athavale A, Abilash V, Hantsbarger G, Geransar P, Lee K, Milicevic S, Perovic M, Raven L, Sajak-Szczerba M, Silber A, Yoon A, and Tozer P contributed to the validation; Athavale A, Abilash V, and Silber A contributed to the visualization; Karki C, Athavale A, Abilash V, Hantsbarger G, Geransar P, Lee K, Milicevic S, Perovic M, Raven L, Sajak-Szczerba M, Silber A, Yoon A, and Tozer P contributed to the writing, review, and editing of the manuscript.
Institutional review board statement: This study was conducted in accordance with the World Medical Association Declaration of Helsinki and Guidelines for Good Pharmacoepidemiology Practices (GPP) and submitted to all applicable local Institutional Review Boards and Ethics Committees to ensure compliance with all ethical standards in each country.
Informed consent statement: Personally identifiable data were not collected in this study. As this was an observational study, consent to any interventional procedure or treatment was not applicable. Consent for participation in the study was solicited by requesting participants to agree to a statement indicating the purpose of the study and a brief summary of the information to be collected. This was carried out prior to entry into the web-enabled questionnaire with a description of the study and its purpose, and responses.
Conflict-of-interest statement: CK is an employee and shareholder of Takeda Pharmaceuticals. AA is an employee of Trinity Life Sciences, commissioned by Takeda Pharmaceuticals to conduct this study. VA is an employee of Trinity Life Sciences, commissioned by Takeda Pharmaceuticals to conduct this study. GH is an employee and shareholder of Takeda Pharmaceuticals. PG is an employee and shareholder of Takeda Pharmaceuticals. KL has served on advisory boards for Takeda Pharmaceuticals. SM is an employee and shareholder of Takeda Pharmaceuticals. MP has no conflicts of interest to disclose. LR has served on advisory boards for Roche and Takeda Pharmaceuticals. MSS has nothing to disclose. AS is an employee of Trinity Life Sciences, commissioned by Takeda Pharmaceuticals to conduct this study. AY is an employee of Takeda Pharmaceuticals. PT has received speaker’s fees from Ferring and Takeda Pharmaceuticals and served on advisory boards for Takeda Pharmaceuticals.
Data sharing statement: Data sets supporting the results from this study are available from the corresponding author upon reasonable request. The data sets will be provided after deidentification, in compliance with applicable privacy laws, data protection, and requirements for consent and anonymization.
STROBE statement: The authors have read the STROBE Statement—checklist of items, and the manuscript was prepared and revised according to the STROBE Statement—checklist of items.
Open-Access: This article is an open-access article that was selected by an in-house editor and fully peer-reviewed by external reviewers. It is distributed in accordance with the Creative Commons Attribution NonCommercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: https://creativecommons.org/Licenses/by-nc/4.0/
Corresponding author: Chitra Karki, Director, Global Evidence and Outcomes-Gastroenterology, Takeda Pharmaceuticals United States, Inc, 350 Massachusetts Avenue, Cambridge, MA 02139, United States. chitra.karki@takeda.com
Received: July 21, 2023
Peer-review started: July 21, 2023
First decision: August 15, 2023
Revised: September 27, 2023
Accepted: October 30, 2023
Article in press: October 30, 2023
Published online: November 27, 2023
Abstract
BACKGROUND

Patients with Crohn’s disease (CD) are at risk of developing complications such as perianal fistulas. Patients with Crohn’s perianal fistulas (CPF) are affected by fecal incontinence (FI), bleeding, pain, swelling, and purulent perianal discharge, and generally face a higher treatment burden than patients with CD without CPF.

AIM

To gain insights into the burden of illness/quality of life in patients with CPF and their treatment preferences and satisfaction.

METHODS

This cross-sectional observational study was conducted in patients with CD aged 21-90 years via a web-enabled questionnaire in seven countries (April-August 2021). Patients were recruited into three cohorts: Cohort 1 included patients without perianal fistulas; cohort 2 included patients with perianal fistulas without fistula-related surgery; and cohort 3 included patients with perianal fistulas and fistula-related surgery. Validated patient-reported outcome measures were used to assess quality of life. Drivers of treatment preferences were measured using a discrete choice experiment (DCE).

RESULTS

In total, 929 patients were recruited (cohort 1, n = 620; cohort 2, n = 174; cohort 3, n = 135). Short Inflammatory Bowel Disease Questionnaire scores were worse for patients with CPF (cohorts 2 and 3) than for those with CD without CPF (cohort 1): Mean score 3.8 and 3.7 vs 4.1, respectively, (P < 0.001). Similarly, mean Revised FI and FI Quality of Life scores were worse for patients with CPF than for those with CD without CPF. Quality of Life with Anal Fistula scores were similar in patients with CPF with or without CPF-related surgery (cohorts 2 and 3): Mean score 41 and 42, respectively. In the DCE, postoperative discomfort and fistula healing rate were the most important treatment attributes influencing treatment choice: Mean relative importance 35.7 and 24.7, respectively.

CONCLUSION

The burden of illness in CD is significantly higher for patients with CPF and patients rate lower postoperative discomfort and higher healing rates as the most desirable treatment attributes.

Keywords: Burden of illness, Crohn’s disease, Discrete choice experiment, Perianal fistulas, Patient-reported outcomes, Treatment preferences

Core Tip: This is the largest known observational study to quantify the burden of illness associated with Crohn’s perianal fistulas (CPF) across multiple countries, utilizing a comprehensive set of outcomes including symptom burden and impacts, and treatment experience, satisfaction, and preferences. This study confirmed that the burden of illness for patients with Crohn’s disease is significantly higher for those with CPF than those without. Patients with CPF rated lower postoperative discomfort and higher healing rates as the most desirable treatment attributes. Assessing patient treatment preferences is key to helping healthcare professionals with clinical management and treatment decisions associated with CPF.